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Inherited Optic Neuropathies
Published in Vivek Lal, A Clinical Approach to Neuro-Ophthalmic Disorders, 2023
Hui-Chen Cheng, Jared Ching, An-Guor Wang, Patrick Yu-Wai-Man
Idebenone is a synthetic, short-chain analogue of ubiquinone, which is responsible for shuttling electrons from complexes I and II directly to complex III (44–46). The current evidence indicates that a subgroup of LHON patients benefits from idebenone and there is a greater likelihood of a positive response when treatment is initiated within the first year of disease onset (44, 47). Idebenone has been approved by the European Medicine Agency (EMA) to treat LHON and the recommended dose is 300 milligrams three times per day (48). Gene therapy based on allotopic expression of wild-type MTND4 is showing promise for LHON patients carrying the m.11778 G>A mtDNA mutation who are treated within 1 year of disease onset (42, 49, 50). Mitochondrial replacement therapy has been developed to prevent the maternal transmission pathogenic mtDNA mutations, but there are concerns regarding the ethical implications and long-term health implications (42, 51). Treatment strategies for other inherited optic neuropathies besides LHON are still in the preclinical phase of development.
Should Genome Editing Replace Embryo Selection Following PGT?
Published in Carlos Simón, Carmen Rubio, Handbook of Genetic Diagnostic Technologies in Reproductive Medicine, 2022
Clinical utilization of genome editing should not be considered until more research has been undertaken to solidify knowledge in areas where important deficiencies currently exist. It is imperative that the eventual application of genome editing succeeds in conferring a significant reduction in the risk of inherited disease for the children born following the procedure. While no medical procedure is entirely risk-free, the likelihood of complications resulting from the editing procedure should be low, particularly considering that the impact may extend to future generations and therefore affect multiple individuals. For use in human reproduction, safety, efficacy, and ethical application are paramount. Applying similar scientific rigor and public engagement as was done in the United Kingdom when considering the clinical introduction of mitochondrial replacement therapy (a process that in some respects can also be considered to introduce a heritable alteration to embryos) will be crucial for determining therapeutic potential and introducing the methodology safely. Nonetheless, even in its current form GE represents a powerful research tool, which can greatly enhance the study of human development, expanding our knowledge in this key area.
‘A Precipice in Time’ – Reproductive Biotechnology
Published in Rosa Maria Quatraro, Pietro Grussu, Handbook of Perinatal Clinical Psychology, 2020
While the moral principles prohibiting cloning are clear cut, those of mitochondrial replacement therapy are less so. As of last year, the UK allowed the creation of ‘three-parent babies’ to prevent diseases being passed on.7 We are just beginning to realise the implications of donated gametes changing the future hereditary germlines of an individual family’s descendants. But CRISPR goes further – it permanently alters distribution of genes in the population. In fact, the immense power of gene editing and the relative ease of its application actually led President Obama’s Director of National Intelligence James Clapper to describe CRISPR as a ‘weapon of mass destruction’ in 2016 (cited by Cobb, 2017).Again, it is a matter of unintended consequences.8 No one is sure what will happen when a gene drive is altered – since its frequency increases exponentially with each generation, thereby rapidly ‘flooding’ the whole population! Many thoughtful writers, philosophers and, indeed, even the inventors of CRISPR, have expressed their anxieties warning that heritable human genetic modifications pose serious risks, and the therapeutic benefits are tenuous (e.g. Lanphier et al., 2015). And they too reiterate the need for global regulation of gene drives (e.g. Baltimore et al., 2015).
Bioethics, (Funding) Priorities, and the Perpetuation of Injustice
Published in The American Journal of Bioethics, 2022
Rachel Fabi, Daniel S. Goldberg
Of course, some may view the funding disparities in these two areas as the natural product of what falls within the scope of bioethics as a field. Narrowly construed, some might argue, bioethics is the study of the ethics of science and medicine. Mitochondrial replacement therapy, that argument may suggest, is clearly within the domain of bioethics. Food insecurity, on the other hand, may be too far afield of bioethics’ purview, straying instead into the murky territory of some other branch of applied ethics. We do not propose to hash out here what is or is not bioethics, but we believe, and are not alone in believing, that the field can and does encompass the ethics of those policies and practices that affect health and other non-health dimensions of well-being. This is in part why Powers and Faden’s nonexceptionalist theory of social justice is so important. Resources needed to address the densely-woven patterns of disadvantage that create insufficiencies in any of the six dimensions of well-being are worth scholarly and—we submit—bioethical attention. This is especially the case when the sources of structural disadvantage drive insufficiencies in health as well as in other essential dimensions of well-being.
Assessing the Ethical Distinctions Between Different Types of Prospective Human Germline Genetic Interventions
Published in The American Journal of Bioethics, 2020
In this country, a 2016 report of an expert committee of the National Academy of Sciences and Institute of Medicine concluded that it was ethically permissible to go forward with mitochondrial replacement therapy in cases where a mother is at risk of transmitting a severe form of mitochondrial genetic disease that could lead to a child’s early death or substantial impairment. As a precaution the committee recommended that in the initial clinical investigations only male embryos be allowed to be the basis of potential pregnancies in order to prevent potential adverse consequences to be passed on to future generations (National Academies of Sciences, Engineering and Medicine 2016). Recently, a group of bioethicists published a letter in support of mitochondrial replacement therapy that asked the FDA to find a path around the rider that Congress has attached to every annual FDA appropriation since 2015 that prohibits the agency from reviewing applications in which a human embryo is modified to include a heritable genetic modification so as to permit review of clinical trials, oversight and data collection regarding mitochondrial replacement therapy (Adashi et al. 2019). I was a cosigner.
It Is Time to Consult the Children: A Mother Who Faced Mitochondrial Replacement and Her Son Consider the Limits of Genetic Modification
Published in The American Journal of Bioethics, 2020
Susan M. Wolf, Jacob S. Borgida
So far, deliberate genetic modification in human reproductive technology has been limited. However, in the late 1990s, a small cohort of women seeking to conceive were offered mitochondrial replacement therapy (MRT) (Kula 2016).1 This technique modifies the genetic makeup of the offspring by creating a child with three genetic forebears—the sperm source, source of the ovum nucleus, and source of the mitochondria, which have their own genomes. This produces a phenomenon not seen in nature, a child with three genetic parents. Other genetic modification of children being conceived has been debated, including somatic gene alteration, historically overseen by the Recombinant DNA Advisory Committee at NIH and the Food and Drug Administration (FDA). Both bodies have refused to consider protocols involving germline genetic alteration, but illicit use of that technology to conceive twin girls in China has prompted an outpouring of ethics guidelines (e.g., Lander et al. 2019).